VLCAD deficiency: pitfalls in newborn screening and confirmation of diagnosis by mutation analysis

Mol Genet Metab. 2006 Jun;88(2):166-70. doi: 10.1016/j.ymgme.2005.12.012. Epub 2006 Feb 20.

Abstract

We diagnosed six newborn babies with very long-chain acyl-CoA dehydrogenase deficiency (VLCADD) through newborn screening in three years in Victoria (prevalence rate: 1:31,500). We identified seven known and two new mutations in our patients (2/6 homozygotes; 4/6 compound heterozygotes). Blood samples taken at age 48-72 h were diagnostic whereas repeat samples at an older age were normal in 4/6 babies. Urine analysis was normal in 5/5. We conclude that the timing of blood sampling for newborn screening is important and that it is important to perform mutation analysis to avoid false-negative diagnoses of VLCADD in asymptomatic newborn babies. In view of the emerging genotype-phenotype correlation in this disorder, the information derived from mutational analysis can be helpful in designing the appropriate follow-up and therapeutic regime for these patients.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acyl-CoA Dehydrogenase, Long-Chain / blood
  • Acyl-CoA Dehydrogenase, Long-Chain / deficiency*
  • Acyl-CoA Dehydrogenase, Long-Chain / genetics*
  • Amino Acid Substitution / genetics
  • Blood Specimen Collection / methods
  • Carnitine / metabolism
  • Carnitine / therapeutic use
  • DNA Mutational Analysis / methods
  • Humans
  • Infant, Newborn
  • Mass Spectrometry
  • Metabolism, Inborn Errors / diagnosis*
  • Metabolism, Inborn Errors / genetics
  • Metabolism, Inborn Errors / metabolism
  • Mutation
  • Neonatal Screening / instrumentation
  • Neonatal Screening / methods
  • RNA Splice Sites / genetics

Substances

  • RNA Splice Sites
  • Acyl-CoA Dehydrogenase, Long-Chain
  • Carnitine